Rare diseases

News

Boehringer's TRPC6 inhibitor apecotrep, formerly developed for COVID-19, has shown efficacy in the rare kidney disease FSGS in a phase 2 trial.

News

An EMA nod for Otsuka's Dawnzera offers EU patients with HAE a drug that can be self-administered by patients with up to eight weeks between doses.

News

Children with a devastating, rare disorder of copper metabolism, called Menkes disease, now have an FDA-approved treatment for the first time.

News

Hutchmed is preparing to file for approval of its drug for the rare disorder wAIHA in China, after chalking up a win in a pivotal phase 2/3 trial.

News

As the celebrations for the end of 2025 gathered pace, the FDA delivered a year-end blow to Corcept by turning down its new Cushing's drug.

News

Two phase 3 trials of Ultragenyx and Mereo's sclerostin inhibitor setrusumab in osteogenesis imperfecta missed their primary efficacy objectives.